Regulatory & Clinical:
During the second quarter, Amicus met with the
With respect to the pivotal study, Amicus will incorporate SAWP feedback on key elements of the nature and design of the pivotal study. The pivotal study is planned to focus on up to 80 ERT-switch patients with 6-minute walk as the primary endpoint and a primary treatment period up to 12 months. Patients enrolled in the current prospective observational study being conducted by Amicus (STRIDE Study 003) would be entered into the pivotal treatment study. This pivotal study is expected to commence in 2H 2018, pending feedback from the Type C meeting with
The SAWP was also supportive of studying additional patient populations, including pediatric Pompe patients and ERT-treatment naïve Pompe patients. Amicus expects to include these patient populations in studies to initiate in 2019, in addition to the pivotal study in ERT-switch patients.
With respect to a pathway for conditional approval, while the SAWP specifically noted that the efficacy data for AT-GAA to date appear “promising” the SAWP indicated that the current clinical package is not sufficient for a Conditional Marketing Authorization Application at this time. Amicus intends to continue a dialogue on a potential pathway for conditional approval with the EMA authorities in 2019 with further data on both efficacy and safety to include:
- Data from up to 10 additional ERT-switch patients in a new Cohort 4 as part of the ongoing Phase 1/2 study (data expected in 2019)
- Presentation of longer-term clinical data out to 18-months for the 19 original Phase 1/2 patients (data expected in 2H 2018)
- Completion of a retrospective natural history study in approximately 100 ERT-treated Pompe patients (data expected in 2H 2018)
As the clinical data continue to develop, Amicus will also be able to commence the Process Performance Qualification (PPQ) runs at the 1,000 Liter (commercial scale). These PPQ manufacturing runs will be essential for any marketing application under any pathway in both the EU and
In a separate meeting with the German regulatory authorities (BfArM), Amicus received scientific advice indicating general agreement with the manufacturing strategy for ATB200, including on the strategy to demonstrate comparability between drug substance and drug product manufactured at the 1,000 liter scale and drug substance and drug product manufactured at the 250 liter scale.
About Scientific Advice:
Scientific Advice is a procedure offered by the EMA to stakeholders for clarification of questions arising during development of medicinal products. The scope of Scientific Advice is limited to scientific issues, i.e. to quality, non-clinical and clinical aspects of the concerned medicinal product not yet unequivocally covered by published scientific guidelines. Scientific Advice is legally non-binding and is based on the current scientific knowledge which may be subject to future changes.
About AT-GAA (ATB200/AT2221)
ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In preclinical studies, ATB200 was associated with increased tissue enzyme levels and reduced glycogen levels in muscle, which was further improved when AT2221 was co-administered with ATB200. Amicus Therapeutics is currently conducting a global Phase 1/2 study (ATB200-02) to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of ATB200/AT2221.
AT-GAA is an investigational product and is not approved by regulatory authorities in any jurisdiction.
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA leads to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Pompe disease can be debilitating, and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to clinical development of our product candidates, the timing and reporting of results from clinical trials and the prospects and timing of the potential regulatory approval of our product candidates. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress and timing of clinical trials, may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the
Associate Director, Investor Relations
Source: Amicus Therapeutics, Inc.